Disease modeling and gene correction of LGMDR21 iPSCs elucidates the role of POGLUT1 in skeletal muscle maintenance, regeneration, and the satellite cell niche

Autosomal recessive limb-girdle muscular dystrophy 21 (LGMDR21) is caused by pathogenic variants in protein O-glucosyltransferase 1 (POGLUT1), which is responsible for O-glucosylation of specific epidermal growth factor (EGF) repeats found in ∼50 mammalian proteins, including Notch receptors. Previo...

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Autori principali:	Jose L. Ortiz-Vitali, Jianbo Wu, Nasa Xu, Annie W. Shieh, Nima Niknejad, Megumi Takeuchi, Carmen Paradas, Chunru Lin, Hamed Jafar-Nejad, Robert S. Haltiwanger, Sidney H. Wang, Radbod Darabi
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2023-09-01
Serie:	Molecular Therapy: Nucleic Acids
Soggetti:	MT: RNA/DNA Editing POGLUT1 LGMDR21 iPSCs gene correction CRISPR-Cas9
Accesso online:	http://www.sciencedirect.com/science/article/pii/S216225312300210X
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